Millions in Grant Funding Provide Foundation of Opportunity

The National Institute of Health aims to supports biomedical science and behavioral research through the pursuit of knowledge of the biology and behavior of living systems and to then apply that knowledge to “extend healthy life and reduce the burdens of illness and disability.” NIH’s own role in this mission includes the provision of funding grants and/or cooperative agreements. To ensure that funds are allocated to organizations aligned with this goal, NIH first determines whether or not the applying company’s project will yield a “sustained, powerful influence on the research field(s) involved.”

Obtaining a grant is much more than a simple petition and business plan. In order for a company to receive a grant from NIH, an applying company’s project must undergo peer review and demonstrate, in addition to other considerations, the following five criteria:

• Significance – address an important problem or critical barrier to progress in the field; Investigators – doctors, collaborators and other researchers must be well-suited, experienced and trained for the project; • Innovation – the application must challenge and seek to shift current research or clinical practice by utilizing novel concepts, approaches, instrumentation or intervention; • Approach – appropriately strategized to accomplish the specific aim of the project; and • Environment – will scientific environment in which the work will be conducted contribute to the probability of success?

The rewards of meeting these criteria are often invaluable. Case in point: VistaGen, Inc., a San Francisco-based stem cell company focused on drug rescue and regenerative medicine backed by a team of stem cell research and development teams and collaborators that for 15 years have focused on controlling the differentiation of pluripotent stem cells to produce multiple types of mature, functional, adult human cells for drug rescue applications.

Since its inception in 1998, the company has received a total of $8.8 million in grant funding from the NIH for phase 1 clinical development of its AV-101 lead small molecule drug candidate.

This funding enabled the company to complete phase 1 development of AV-101, an orally available small molecule prodrug candidate designed to address needs in the multi-billion dollar neurological disease disorders market, such as neuropathic pain, epilepsy and depression. VistaGen has submitted an AV-101 IND application with the U.S. FDA to cover clinical development for neuropathic pain, though the company believes that completed phase 1 AV-101 safety studies will also support development of AV-101 for multiple indications, including epilepsy and depression.

VistaGen’s plan, contingent upon completion of this offering, is to pursue potential opportunities for further development and commercialization of AV-101 on a stand-alone or corporate partnership basis. If successful, the company says it intends to use the net proceeds from such an arrangement to expand its drug rescue and regenerative medicine programs, which are based on its stem cell technology platform, Human Clinical Trials in a Test Tube™.

Receiving NIH funding marked a pivotal moment in VistaGen’s history, providing the company with a monetary avenue to pursue its broader mission to commercialize therapeutically and commercially promising regenerative medicine programs.